A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy Volunteers
Phase 1
Completed
- Conditions
- TTR-mediated Amyloidosis
- Interventions
- Drug: ALN-TTRSC (revusiran)Drug: Sterile Normal Saline (0.9% NaCl)
- Registration Number
- NCT01814839
- Lead Sponsor
- Alnylam Pharmaceuticals
- Brief Summary
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC (revusiran) in healthy volunteer subjects.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 85
Inclusion Criteria
- Body mass index (BMI) must be ≥ 16.5 kg/m2 and ≤ 35.0 kg/m2;
- Female subjects must be of non-childbearing potential; e.g., postmenopausal or pre-menopausal with surgical sterilization;
- Male subjects agree to use appropriate contraception;
- Adequate blood counts, liver and renal function;
- Non-smokers for at least 3 months;
- Willing to give written informed consent and are willing to comply with the study requirements;
- Subject was born in Japan and has lived outside of Japan for <10 years, and subject's biological parents and grandparents are fully Japanese and were born in Japan (cohorts 19 and 20).
Exclusion Criteria
- Known human immunodeficiency virus (HIV) positive status or known or suspected systemic bacterial, viral, parasitic, or fungal infection;
- Subjects with a history of multiple drug allergies or intolerance to SC injection;
- History of drug abuse and/or alcohol abuse;
- Receiving an investigational agent within 3 months prior to study drug administration;
- Considered unfit for the study by the Principal Investigator.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description ALN-TTRSC (revusiran) ALN-TTRSC (revusiran) - Sterile Normal Saline (0.9% NaCl) Sterile Normal Saline (0.9% NaCl) -
- Primary Outcome Measures
Name Time Method The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation. Up to 63 days
- Secondary Outcome Measures
Name Time Method Pharmacokinetics (PK) of ALN-TTRSC (revusiran) (Cmax, tmax, t1/2, AUC0-last, CL). Up to 90 days Effect of ALN-TTRSC (revusiran) on transthyretin (TTR) (Determination of % Lowering of TTR to pretreatment/Baseline Levels). Up to 90 days Effect of ALN-TTRSC (revusiran) on retinol binding protein (RBP) (Determination of % Lowering of RBP to pretreatment/Baseline Levels). Up to 90 days Effect of ALN-TTRSC (revusiran) on vitamin A (Determination of % Lowering of vitamin A to pretreatment/Baseline Levels). Up to 90 days
Trial Locations
- Locations (1)
Clinical Site
🇬🇧London, United Kingdom